Hereditary angioedema (HAE) is a rare genetic disorder characterized by painful, debilitating, and potentially fatal swelling attacks. Lanadelumab is approved as long-term prophylaxis (LTP) in patients with HAE. However, real-world data on LTP use in patients with HAE are limited. To describe clinical characteristics, attack history, and quality of life (QoL) of patients with HAE type I/II who were receiving lanadelumab or other LTPs. Data were drawn from the Adelphi HAE Disease Specific Program, a cross-sectional survey of HAE physicians conducted in the United States from July to November 2021. Physician-reported disease characteristics, HAE attack frequency, and QoL were compared among patients receiving lanadelumab or other LTPs for at least 12 months. Physicians reported data for 144 patients, of whom 29 had received lanadelumab and 115 had received another prophylaxis for at least 12 months. The mean ± standard deviation number of attacks in the previous 12 months was lower among patients receiving lanadelumab than other LTPs (2.3 ± 3.1 versus 3.4 ± 2.8, respectively; p = 0.075). Although both groups had similar current disease activity and severity, more patients receiving lanadelumab versus other LTPs had high disease activity (51.7% versus 12.5%, respectively; p < 0.0001) and disease severity rated as severe (51.7% versus 16.1%, respectively; p = 0.0001) at diagnosis. Physicians reported that more patients who received lanadelumab had good or very good QoL (72.4%) than those receiving other LTPs (36.5%) (p = 0.003). Analysis of these findings suggests lower attack frequency, lower symptomatic impact, and better QoL in patients treated with lanadelumab than another prophylaxis in a real-world setting.
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http://dx.doi.org/10.2500/aap.2024.45.240046 | DOI Listing |
Pediatr Allergy Immunol
December 2024
French National Reference Center for Angioedema (CREAK), Center of Excellence and Reference (ACARE), Internal Medicine Department, Grenoble Alpes University Hospital, Grenoble, France.
Background: Hereditary angioedema (HAE) in children has specific features and requires multidisciplinary management.
Methods: We performed a literature search and underwent in-depth discussions to provide practical tools for physicians.
Results: HAE is a rare, life-threatening genetic disorder.
Allergol Select
November 2024
Institute of Allergology, Charité Universitätsmedizin Berlin und Fraunhofer Institute for Translational Medicine and Pharmacology ITMP, Allergology and Immunology, Berlin.
Allergol Select
November 2024
Department of Oto-Rhino-Laryngology. Head and Neck Surgery, Ulm University Medical Center, Ulm.
Clin Rev Allergy Immunol
December 2024
Grenoble Alpes University, CNRS, UMR 5525, VetAgro Sup, Grenoble INP, National Reference Center for Angioedema (CREAK), CHU Grenoble Alpes, TIMC, Grenoble, France.
Front Immunol
October 2024
Internal Medicine Department, Fatebenefratelli Hospital, Milan, Italy.
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